Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02303626 | 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks | PHASE2 | COMPLETED | 110 | — | — | Dec 17, 2014 | Jan 1, 2016 | Nov 17, 2025 | 40 | United States, Belgium +6 |
| NCT01984788 | Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE | PHASE2 | COMPLETED | 24 | — | — | Nov 1, 2013 | May 1, 2014 | Aug 18, 2014 | 6 | Germany, United Kingdom |
| NCT02218294 | Study to Determine How BCX4161 is Metabolized and Eliminated by the Body | PHASE1 | COMPLETED | 7 | — | — | Aug 1, 2014 | Sep 1, 2014 | Oct 29, 2014 | 1 | United Kingdom |
| NCT02125162 | A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161 | PHASE1 | COMPLETED | 28 | — | — | Apr 1, 2014 | Jul 1, 2014 | Aug 15, 2014 | 1 | United Kingdom |
An angioedema attack was defined as swelling at any location reported by participant, which had no swelling earlier. Total number of confirmed attacks during the treatment period standardized to a weekly attack-rate to adjust for the total duration of treatment. The attack rate was derived for each participant by treatment period. The weekly attack rate was equal to the total number of confirmed attacks during a treatment period divided by the duration of the treatment (in days) times 7 days.
| Arm | Type | Description |
|---|---|---|
| BCX4161 300 mg three times daily | EXPERIMENTAL | Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth |
| BCX4161 500 mg three times daily | EXPERIMENTAL | Five BCX4161 capsules (100 mg) to be taken three times daily by mouth |
| Placebo three times daily | PLACEBO_COMPARATOR | Five placebo capsules to be taken three times daily by mouth |
| BCX4161 | ACTIVE_COMPARATOR | 400 mg TID for 28 days |
| Placebo | PLACEBO_COMPARATOR | TID for 28 days |
| [14C] BCX4161 | EXPERIMENTAL | Includes a radiolabelled dose of \[14C\] BCX4161 and unlabelled BCX4161 |
| Treatment A | EXPERIMENTAL | BCX4161 400 mg formulated as hard gelatin capsules given orally under fasting conditions x1 |
| Treatment B | EXPERIMENTAL | BCX4161 400 mg formulated as soft gelatin capsules given orally under fasting conditions x1 |
| Treatment C | EXPERIMENTAL | BCX4161 400 mg formulated as soft gelatin capsules given orally after a high-fat breakfast x1 |
| Name | Type | Description |
|---|---|---|
| BCX4161 | DRUG | - |
| Placebo | DRUG | - |
Key Inclusion Criteria: 1. A clinical diagnosis of HAE type I or II 2. Documented HAE attacks within a defined calendar period; in the absence of documented HAE attacks, participants will be required to enter a run-in period to document attacks 3. Access to acute attack medications 4. Sexually acti...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration, Biological NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat, berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| KalVista Pharmaceuticals, Inc. | KALV | 1 | PHASE3 | KVD900, Drug: KVD900 |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |